AFTD hosted an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting on March 5, allowing persons diagnosed and current and former caregivers a valuable one-time opportunity to inform the development of federal regulatory processes for potential FTD therapeutics.

An estimated 550 people joined the interactive livestream, hosted by AFTD CEO Susan L-J Dickinson and James E. Valentine, who previously worked with the FDA.

“To the people and families from around the world who live the FTD journey every day, this meeting is for you,” Dickinson said during the meeting. “It’s your chance to ensure that the FDA understands the urgent need for FTD treatments, which symptoms you find the most burdensome, and what you would want most from an approved therapy.”

EL-PFDD meetings help to ensure that the experiences, perspectives, needs, and priorities of persons affected by a specific disease are captured and meaningfully incorporated into the FDA’s drug-regulation process. These meetings help the FDA to understand what it’s like to live with a particular disease with an insider’s perspective of its symptoms and how they affect daily living.

The AFTD-led PFDD meeting featured live input from persons who are either diagnosed with FTD themselves or who are current or former caregivers. Participants answered live FTD-related polling questions; many called in to share personal stories of themselves or loved ones. Viewers were also able to send in written comments about their experiences.

Following the EL-PFDD meeting, AFTD will prepare a report summarizing these testimonies. Known as a “Voice of the Patient” report, these summaries are a key resource for FDA regulators to help better understand what people living with FTD and their care partners/ caregivers most want to see in treatment options.

The Voice of the Patient Report will be publicly available on AFTD’s website for review.