SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA

Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to testify directly to the FDA.

The PFDD meeting will include testimony on a variety of topics important to our community, including:

  • The impact of SMA on daily life
  • What would amount to meaningful change in SMA patients’ everyday lives
  • Benefit/risk
  • Our priorities in drug development
  • Our preferences and expectations for an improved treatment

The testimony will also be used to create a Voice of the Patient report. This report will become part of the review process for all SMA treatments that are submitted the FDA.

The meeting is tentatively scheduled for spring 2017. We will pass along the exact date when it is finalized. We will also be holding a webinar in early 2017 on FDA Interactions. The webinar will cover how to participate in the PFDD meeting as well as other opportunities for engagement with the FDA.

Leading the Way in FDA Engagement

Earlier this year, Cure SMA, along with our partners at MDA and the SMA Foundation, gathered the information and prepared a formal request for this PFDD meeting. We thank our partners for their hard work in making this meeting possible.

In addition, we will be announcing a new community survey in November. This survey will cover a number of areas that are relevant to the real-world experiences of living with SMA. Through this survey, we will collect and distribute data to support the testimony presented at the PFDD meeting.

At this very critical time, many of you have asked us how you can participate in the process. Participating in this survey and, if you are able, participating in the PFDD meeting, will provide a tangible and measurable impact for our whole community.

For More Information

Read an update from earlier this year on how we’ve been working to engage the FDA with our community.

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