The TSC Alliance sponsored a patient-focused drug development meeting (PFDD) with the FDA on June 21, 2017. The purpose of this meeting was for individuals affected by TSC and caregivers of dependent adults or children to communicate their perspectives on living with TSC to the FDA.

The TSC PFDD meeting was divided into two parts. The morning focused on input from parents of children with TSC who have experienced, or are at risk for developing, epilepsy. The afternoon focused on adults with TSC and/or LAM who have experienced, or are at risk for developing, angiomyolipomas or LAM. The TSC 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.

In late October 2017, the TSC Alliance submitted this meeting’s Voice of the Patient report to the FDA. This report provides a detailed summary of the patient testimony presented at the meeting and communicates needs and improvements patients want and hope to see in their daily lives.