LAM and TSC Patients Present to the FDA

While in Washington, DC in June, The LAM Foundation and TS Alliance joined forces to communicate the patient perspective on living with LAM to the Food and Drug Administration (FDA). This important work helps the FDA understand the ‘Voice of the Patient’ and gives them perspective on living with these diseases so they can make better decisions on future drug development.

This meeting was attended by patients and their care givers, clinicians and members of the FDA. In total, over 60 people attended in person while more than 100 others participated via a live webcast. Women with LAM, along with individuals with TSC and caregivers of individuals with TSC gave testimonies to share their experiences living with this disease. Following the testimonies, all those in attendance and those participating online were given the opportunity to give their thoughts.

Frank McCormack, MD and Martina Bebin, MD, MPA presented clinical overviews of LAM and TSC, respectively, and explained different manifestations of each disease. The audience also had the pleasure of hearing from Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research and Martha Donoghue, MD, Acting Associate Deputy Director, Division of Oncology Products from the FDA speak about the importance of Patient-Focused Drug Development meetings and how they are helping the FDA be more efficient during the drug approval process.

Thank you to all those who attended in person or online, those who spoke at the meeting and everyone who helped to make this meeting a success. A “Voice of the Patient” report is currently being written from this information gathered at this meeting and will be filed with the FDA to be used as a reference for any future drug development associated with LAM or TSC.