SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017

Tuesday, April 18, 2017, has been announced as the date for the SMA 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 share their stories with the FDA on a variety of topics important to our community.

Members of the community will be able to participate in two ways. A limited number of in-person spots are available on a first-come, first-served basis. There will also be a live-streaming webcast for those unable to travel to the meeting. Registration for both the in-person and webcast options will launch within a few weeks, and all participants will be able to contribute to the open comment portions of the meeting.

With the first ever approval of a treatment for SMA, and many additional therapies in development and in clinical trials, this is an important time to continue our work with the FDA. Factors that influence the treatment and management of SMA—such as benefit/risk, the design of future clinical trials, and expectations for future therapies—will continue to evolve as treatment evolves, and it is critical that the FDA understand the growing and changing needs of our community.

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

In early spring, we will be holding a webinar on the interactions between the FDA and the SMA community. This webinar will include additional information on the upcoming PFDD meeting. A date for the webinar will be announced later this month.

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