FDA Engagement Update: The Voices of SMA

By Cure SMA | Published On September 23, 2015

FDA Engagement Update: The Voices of SMA

Thanks to our vibrant community, August was one of our largest and best awareness months ever, with unprecedented levels of public awareness and outreach for SMA.

Toward the end of August, we challenged our community to seize this momentum, and convert awareness into action and advocacy.

One of these opportunities to convert awareness into action was our "Voices of SMA" project—an outreach to the FDA in order to educate them about:

  1. Our community’s priorities in drug development
  2. The impact of SMA on patients and their families’ daily lives
  3. What would amount to meaningful change in SMA patients’ everyday lives, and
  4. Our community’s preferences and expectations for an improved treatment.

As we expected, our community came through with dozens of stories that eloquently, gently yet pointedly, illustrated these concepts for the FDA.

With these stories, we created The Voices of SMA, a booklet summarizing the key themes, and highlighting those same themes through selected excerpts from the stories. This booklet, along with a complete collection of all the stories submitted, was distributed to all the top decision-makers at the FDA.

As part of the PFDD (patient-focused drug development) initiative, these stories will become part of the review process if and when an SMA drug is brought forward for approval.

Members of FDA leadership have extended their thanks to the Cure SMA community for sharing their stories, calling them "very moving and informative" and full of "important insights." Your voices have made a difference!

The Voices of SMA is just one of the critical next steps in our FDA Engagement Initiative. In the coming weeks, we’ll be announcing other next steps as we continue to move forward toward potential FDA approval for the SMA drugs currently in development.

Topics: Community & Awareness, Advocacy, Front Page News

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