Individuals & Families

Patient-Focused Drug Development

Tuberous Sclerosis Complex Patient-Focused Drug Development Meeting

The TS Alliance sponsored a patient-focused drug development meeting (PFDD) with the FDA on June 21, 2017, at the Hyatt Regency on Capitol Hill in Washington, DC, from 8:30 a.m. to 4:30 p.m. 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. This meeting was free and open to the public, and it was webcast live online.

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.

Voice of the Patient Online Survey

To ensure the perspectives of all affected individuals, family members, or caregivers are represented, additional comments are invited from the public. Please take a few minutes to complete this online survey by July 25. Responses will be grouped, analyzed, and presented to the US Food and Drug Administration (FDA) in a Voice of the Patient report the TS Alliance will submit by the end of 2017. The report will be used when FDA considers approving a new drug for TSC or LAM.

PFDD Videos

If you were unable to attend the PFDD meeting in person or online, you can view recordings of the meeting below:

Morning Session: Infants with TSC, Part 1

Morning Session, Infants with TSC, Part 2

Afternoon Session: Adults with TSC and/or LAM, Part 1

Afternoon Session: Adults with TSC and/or LAM, Part 2