The TSC Alliance has just submitted the Voice of the Patient report to the US Food and Drug Administration (FDA). This report summarizes the Patient-Focused Drug Development (PFDD) meeting that we held on June 21. The PFDD meeting was designed to communicate to FDA the impacts of TSC on individuals’ daily lives, what types of treatment benefits make the most impact on peoples’ lives, and individuals’ and caregivers’ perspectives on how well available therapies are working. The report is now available on our PFDD page.


In addition to summarizing the meeting, the report also includes the results of our international drug development survey for TSC and LAM. This survey was conducted in English, Spanish, and French, and received over 1,300 responses from 57 countries. The quantitative data complement the rich personal stories that were recorded during the event.