Individuals & Families

Patient-Focused Drug Development

Tuberous Sclerosis Complex Patient-Focused Drug Development Meeting

The TSC 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 Report

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.

In addition, you can read the cover letter to Dr. Janet Woodcock and others at the FDA submitted with this report.

Letters to FDA Documenting Unmet Medical Needs

PFDD Transcripts

Click the links below to read transcripts from the PFDD meeting:

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